Medical Management of Marfan Syndrome
44:21-48:07
I'm just going to go back and say that we were able to explore, you know, one of the questions was, you know, was there evidence that these medications reduced the rate of tearing of the aorta and things like surgery and that, that kind of thing and, and the answer to that question and, and this, it may be difficult for people to understand but I, I'll explain it the best way I can.
ARBやβ遮断薬によって大動脈解離や大動脈手術を避けられるかどうかについて、こちらのスライドを使って、できる限りわかりやすく説明したいと思います。
The, the, the answer is that this may be surprising. There were not enough patients thankfully having aortic tears or aortic surgery or dying in these trials and, and this is a positive message I think for everyone on this call. There were not enough patients having aortic tears or having surgeries or dying thankfully in these trials for us to draw any meaningful conclusions about whether these medications impact on these things or not, okay? and that's because these patients were diagnosed, they were being followed carefully by experts who knew what they were doing in, in good centers in America and Canada and in, in the UK and in the, in other European countries, they were compliant with their medications, they were on good medications like beta blockers and ARBs, they were having regular echoes, they were having surgery when surgery was necessary and they were only being followed over a short number of years, two, three, four, five years and that's a very positive message because look, look here, you know, this, this is, this is data from 1,500 patients approximately who were followed over three, four, five years and in the, in the patients given ARBs there were zero deaths. There were zero aortic dissections. What a fantastic message for you guys, you know, so I, I, I, you know, that's in the ARB versus control trials, so I think this sends a very optimistic message.
回答としては、これらの臨床試験においては、大動脈解離や大動脈手術、死亡といった事象は多くなかったということになります。皆さんにとっては、前向きなニュースといえます。大動脈イベントに対する薬の影響について、意味ある結論を下すには、大動脈解離などを起こした患者さんの数が足りなかった、という結果です。その理由としては、患者さん達は、米国やカナダ、英国、欧州の実績のある医療施設において診断を受け、治療に詳しい専門医に慎重にフォローされ、ARBやβ遮断薬といった薬のガイドラインに則った投薬治療や定期的な心エコー検査を受け、手術が必要なタイミングで手術を受けていたからです。これらの患者さんの追跡期間は短く、2、3、4、5年でした。こちらの表は、1,500名ほどの患者さんを対象として、3〜5年追跡調査したものですが、ARBを飲んでいた患者さんについては、死亡と大動脈解離は0件でした。これらはARBとプラセボの比較試験から得られたもので、楽観的に考えられる結果です。
Some people have tried to use this information to suggest that, that the, the drugs don't reduce these clinical outcomes but that is a misunderstanding or, or a misinterpretation of how to interpret meta-analyses, I'm afraid to say and I do not agree with it. The, the the goal of the meta-analysis from the outset the primary outcome of the meta-analysis was to look at whether it reduced the rate of enlargement of the aortic root, which is a very, very important measure in Marfan syndrome. If I had Marfan syndrome I would not want my aortic root expanding rapidly because that's how we decide whether to do aortic surgeries and it's, it's the it's the, best measurement we've got for deciding whether you are in trouble if you've got Marfan syndrome and it's how we decide whether you have surgery or not and it, you know, what I try to do with patients with Marfan syndrome is I, I try not to let them get into a situation where they aorta to get to these dangerous levels and it looks to me like my colleagues who were running these trials in, in the in, in these, in these countries were doing a fantastic job of that by and large and so they should and, and, and that, that is not and so there were not enough events in order to make reliable conclusions about whether the drugs impact on these clinical events. I, I hope I've explained that as well as I can, okay?
この結果について、薬で大動脈イベントが減らないのではないか、と主張する方も一部いらっしゃいますが、メタアナリシスの解釈に誤りがあると思います。このメタアナリシスの主な目標は、薬によって、大動脈基部の拡張速度を遅くすることができるかどうかを調べることでした。この指標はマルファン症候群では非常に重要といえます。私自身がマルファン症候群だとしたら、大動脈基部が急激に拡張してほしいとは思いません。大動脈基部の拡張速度は、手術をするかどうかの基準として用いられ、マルファン症候群の患者さんで問題が起きているかどうかを確かめるための最良の指標ともなっているからです。私はマルファン症候群の患者さんには、大動脈が危険な水準まで拡張しないようにするための治療を行なっていますし、これらの臨床試験を行った他の国の先生方も、全体として、素晴らしい診療を行なっていると思われます。こうした背景があり、大動脈イベントに関する薬の効果について、発生件数が足りず、信頼に足る結論が得られなかったということです。
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
